Gene therapy drug Glybera enters the EU market

November 2, 2012, the European Medicines Agency for Medicinal Products (EMA) adopted a positive decision on the admission of Glybera drug to the EU market. The drug has been developed by Dutch biotechnology company uniQuro and designed for gene therapy of a rare genetic disease - lipoprotein lipase deficiency (LPLD).

The cost of the drug for a single use will be approximately $1.6M. It is expected that the drug will come to the market in the second half of 2013.

LPL enzyme contributes to the breakdown of fat ingested with food. Due to mutations in the gene that codes the production of the enzyme lipase, it is not produced in the right quantity. Such patients are forced to adhere to a very strict diet. Some patients suffer from life-threatening attacks of acute pancreatitis that occur even without breaking the diet.

Glybera is gene therapy drug based on adeno-associated virus (AAV), the genome of which includes LPL gene. AAV feature is the ability to integrate its genome into the host cell genome.

J?rn Aldag, the CEO of uniQuro: The high price of the drug is partly justified by the narrow market. LPL is diagnosed in 1-2 people per million. There are several hundred of such people in the European Union.

Glybera is the world's fourth officially approved gene therapy drug. The first country to permit the use of drugs for gene therapy was China. This country officially permitted two gene therapy drugs in clinical practice in 2003 - Gendicine and Oncorine developed by SiBiono GeneTech Co for the treatment of severe head and neck cancer. Russian company OJSC Human Stem Cell Institute received marketing authorization for gene therapy drug Neovaskulgen in December 2011. The drug is for the treatment of lower limb ischemia. The drug is included in the RF register of medicinal products for medical use and went on sale in October, 2012.